Pathogenesis-based therapies in ichthyoses.

ABSTRACT: During the past 20 years, tremendous progress has been made in our understanding of the molecular basis of many genetic skin conditions. The translation of these laboratory findings into effective therapies for affected individuals has been slow, however, in large part due to the risk of carcinogenesis from random viral genomic integration and the lack of efficacy of topically applied genetic material and most proteins. As intervention at the gene level still appears remote for most genetic disorders, increased knowledge about the cellular and biochemical pathogenesis of disease allows specific targeting of pathways with existing and/or novel drugs and molecules. In contrast to the requirement for personalization of most gene-based approaches, pathogenesis-based therapy is pathway specific

SUBMITTER: Lai-Cheong JE 

PROVIDER: S-EPMC4498576 | biostudies-literature | 2013 Jan-Feb

REPOSITORIES: biostudies-literature